R&D pipeline
These programs are investigating treatments or outcomes that have not all received approval from a health authority. The information presented is not intended to convey conclusions of safety or efficacy. There is no guarantee that the outcome of these studies will result in approval by a health authority.
Vertex is focused on discovering, developing and producing innovative medicines so people with serious diseases can lead better lives. Our scientists don’t see the impossible as an obstacle; they see it as a good place to start.
We have a unique way of building our drug discovery programs to maximize their chances of creating therapies that may dramatically improve patients’ lives. Our focus is on serious diseases where we could truly have a transformative impact for patients, not just an incremental benefit.
We work only on projects where we have a deep understanding of the underlying cause of disease in humans. Then we research and develop therapeutic approaches that are most likely to succeed. Rather than looking for problems we can solve with only the tools we’ve used before, we figure out the problems that need to be solved for the diseases we’re going after and invent the tools to potentially fix them.
These programs are investigating treatments or outcomes that have not all received approval from a health authority. The information presented is not intended to convey conclusions of safety or efficacy. There is no guarantee that the outcome of these studies will result in approval by a health authority.
Vertex is focused on discovering, developing and producing innovative medicines so people with serious diseases can lead better lives. Our scientists don’t see the impossible as an obstacle; they see it as a good place to start.
We have a unique way of building our drug discovery programs to maximize their chances of creating therapies that may dramatically improve patients’ lives. Our focus is on serious diseases where we could truly have a transformative impact for patients, not just an incremental benefit.
We work only on projects where we have a deep understanding of the underlying cause of disease in humans. Then we research and develop therapeutic approaches that are most likely to succeed. Rather than looking for problems we can solve with only the tools we’ve used before, we figure out the problems that need to be solved for the diseases we’re going after and invent the tools to potentially fix them.
Cystic fibrosis
We are submitting regulatory filings globally for geographic expansion and/or label expansions. For information about ongoing clinical studies in the U.S., visit the clinical trials website. For information about non-U.S. sites, visit clinicaltrials.gov.
We are submitting regulatory filings globally for geographic expansion and/or label expansions. For information about ongoing clinical studies in the U.S., visit the clinical trials website. For information about non-U.S. sites, visit clinicaltrials.gov.
We are submitting regulatory filings globally for geographic expansion and/or label expansions. For information about ongoing clinical studies in the U.S., visit the clinical trials website. For information about non-U.S. sites, visit clinicaltrials.gov.
We are submitting regulatory filings globally for geographic expansion and/or label expansions. For information about ongoing clinical studies in the U.S., visit the clinical trials website. For information about non-U.S. sites, visit clinicaltrials.gov.
We are submitting regulatory filings globally for geographic expansion and/or label expansions. For information about ongoing clinical studies in the U.S., visit the clinical trials website. For information about non-U.S. sites, visit clinicaltrials.gov.
We are investigating VX-522, a CFTR mRNA that can be delivered to the lung by lipid nanoparticles to address the underlying cause of CF lung disease in approximately 5,000 people with CF who do not make any CFTR protein that responds to a CFTR modulator therapy.
For information about ongoing clinical studies in the U.S., visit the clinical trials website. For information about non-U.S. sites, visit clinicaltrials.gov.
We’re investigating a portfolio of other small molecule medicines targeting the underlying cause of cystic fibrosis with the aim of achieving carrier levels of CFTR function. This includes CFTR potentiators, which are designed to keep CFTR proteins at the cell surface open longer to improve the flow of salt and water across cell membranes, helping to hydrate and clear mucus from the airways. We’re also researching additional small molecules to address the trafficking and processing defect of the CFTR protein to enable it to move through cells and reach the surface.
We’re investigating potential treatments for people with cystic fibrosis who do not make any CFTR protein. We are investing in our own science and with our external partners CRISPR, Arbor and Moderna to develop other potential approaches with an aim to treat approximately 5,000 people who do not make any CFTR protein that responds to a CFTR modulator therapy. Our efforts to discover and develop therapies to reach as many people as possible with cystic fibrosis through alternative investigational technologies, like mRNA therapies, have been underway for years.
We continue to discover, research and develop a portfolio of small molecules as potential nonopioid medicines for the treatment of acute pain.
We continue to discover, research and develop a portfolio of small molecules as potential nonopioid medicines for the treatment of neuropathic pain.
We continue to discover, research and develop a portfolio of small molecules as potential nonopioid medicines for the treatment of neuropathic pain.
We continue to discover, research and develop a portfolio of small molecules as potential nonopioid medicines for the treatment of acute and neuropathic pain.
Sickle cell disease
We are submitting regulatory filings globally for geographic expansion. For information about ongoing clinical studies in the U.S., visit the clinical trials website. For information about non-U.S. sites, visit clinicaltrials.gov.
We’re investigating small molecule medicines aimed at the underlying cause of sickle cell disease.
Beta thalassemia
We are submitting regulatory filings globally for geographic expansion. For information about ongoing clinical studies in the U.S., visit the clinical trials website. For information about non-U.S. sites, visit clinicaltrials.gov.
We’re investigating small molecule medicines aimed at the underlying cause of transfusion-dependent beta thalassemia.
We continue to research a portfolio of small molecules as potential medicines for the treatment of alpha-1 antitrypsin deficiency.
We are investigating inaxaplin, formerly known as VX-147, aimed at inhibiting high-risk variants of APOL1.
In addition to investigating candidate medicine inaxaplin, formerly known as VX-147, we continue to research and develop a portfolio of small molecule inhibitors for the potential treatment of APOL1-mediated kidney disease.
We are investigating VX-407 for the treatment of ADPKD in patients with a subset of PKD1 genetic variants, with the aim of restoring function to the polycystin 1 (PC1) protein.
We are investigating povetacicept (ALPN-303) with the goal of targeting the underlying cause of IgA nephropathy (IgAN).
We are investigating novel approaches to treating Duchenne muscular dystrophy with the goal of restoring near-full length dystrophin protein expression by targeting certain mutations in the dystrophin gene that cause the disease.
We are investigating VX-670, an oligonucleotide approach using an intracellular Endosomal Escape Vehicle (EEV™) aimed at targeting the underlying cause of myotonic dystrophy type 1.
VX-880 is an investigational allogeneic stem cell-derived, fully differentiated, insulin-producing islet cell therapy manufactured using proprietary technology. It is being evaluated for patients who have type 1 diabetes with impaired hypoglycemic awareness and severe hypoglycemia. VX-880 is being investigated as an infusion into the hepatic portal vein and requires immunosuppressive therapy to protect the islet cells from immune rejection.
VX-264 is an investigational approach encapsulating cells in a protective device to be surgically implanted in the body. VX-264 is being evaluated without the use of immunosuppressive therapy as the devices are designed to shield the cells from the body's immune system.
In January 2017, Vertex entered into a licensing agreement with Merck KGaA, Darmstadt, Germany for the worldwide development and commercialization of four promising research and development programs for the treatment of cancer. As part of the agreement, Merck KGaA, Darmstadt, Germany licensed two clinical-stage programs comprised of the compounds VX-970, VX-803 and VX-984, targeting DNA damage and repair, along with two additional novel research programs that include one immuno-oncology program and a program against a completely novel target. Learn more.
In January 2017, Vertex entered into a licensing agreement with Merck KGaA, Darmstadt, Germany for the worldwide development and commercialization of four promising research and development programs for the treatment of cancer. As part of the agreement, Merck KGaA, Darmstadt, Germany licensed two clinical-stage programs comprised of the compounds VX-970, VX-803 and VX-984, targeting DNA damage and repair, along with two additional novel research programs that include one immuno-oncology program and a program against a completely novel target. Learn more.
In January 2017, Vertex entered into a licensing agreement with Merck KGaA, Darmstadt, Germany for the worldwide development and commercialization of four promising research and development programs for the treatment of cancer. As part of the agreement, Merck KGaA, Darmstadt, Germany licensed two clinical-stage programs comprised of the compounds VX-970, VX-803 and VX-984, targeting DNA damage and repair, along with two additional novel research programs that include one immuno-oncology program and a program against a completely novel target. Learn more.